Families that Participate in Research Help Improve Health for All

Published on in CHOP Family News

For Grace Ebersberger, 18, coming to Children’s Hospital of Philadelphia (CHOP) for a research study has two things going for it: “I like that it helps other people,” she says, “and they pay me.”

Grace’s participation in research has had nothing to do with the numerous orthopaedic surgeries she’s had over the years. Instead, she has participated in a series of studies for the Center for Applied Genomics and the Division of Neurology that started with donating a blood sample when she was 8 and have included several lengthy interviews, answering questions while in an MRI, and playing computer games to track her brain’s processing speed.

Families like the Ebersbergers are critical for CHOP to fulfill its mission of discovering breakthroughs for more effective treatments and, hopefully, cures.

While some research takes place exclusively in the lab, many studies, called clinical trials, need children to participate. “The No. 1 barrier to success of a research project is recruiting qualified participants,” says Chris Gantz, Manager, Recruitment Enhancement Core. “It can make or break a study.”

Gantz acts like a matchmaker. When researchers at CHOP are ready to recruit children and families for a research study, they contact him. He seeks families with children who meet the study’s criteria and connects them to the researchers.

Some studies compare a new medication or intervention for a specific disease against the current standard of care to see which delivers the best outcomes. Other research focuses on uncovering the causes of disease, which can lead to targeted treatment or disease prevention.

Some researchers study how to prevent injuries or how physicians can deliver more effective care by using electronic health records. At a place as big as CHOP, there are hundreds of active studies going on all the time.

In addition to children who have a particular condition, many studies need well children to serve as “healthy controls” so researchers can compare healthy children to patients with a specific disease. Similar to Grace’s experience, children are asked to give blood or have an MRI, for example, and their results are matched against patients of the same gender and age. Participants are usually reimbursed for travel expenses and parking and sometimes receive a small stipend.

In addition to launching the Recruitment Enhancement Core, CHOP created the Clinical Research Finder, a searchable database to make it easier for families to know what current studies are recruiting. You can follow Children’s Hospital on social media to see posts that call for participants. Families can also learn about studies from their child’s doctors and nurses.

When Emily Herrera’s acute lymphoblastic leukemia (ALL) didn’t respond to traditional chemotherapy, she had genetic testing that revealed she had a high-risk type of ALL called Philadelphia-like. When asked by her oncologist, Sarah Tasian, MD, Emily, now 19, agreed to join a clinical trial testing a new medication called ruxolitinib with usual chemotherapy, a treatment researchers believe will work on her type of ALL.

She is the first CHOP patient to take ruxolitinib as part of a larger, multi-institution study from the Children’s Oncology Group. “I wake up every morning happy to take my ‘genetic pill,’ and I’d encourage other patients to take part in clinical trials,” she says.

Reciprocal benefits

Studies at the Center for Autism Research (CAR) “have been a real benefit to us,” says Jennifer Nolte, mom to 9-year-old triplets Erik, Luke and Julia. Both Erik and Luke, who have autism spectrum disorder, have participated in CAR studies.

“They’ve had the opportunity to be in cutting-edge studies — all the latest advances like studying visual and motor acuity — and we end up with extra information we wouldn’t otherwise have,” she says. “It’s very reciprocal: We help the researchers and they help us.”

Another tangible benefit is that volunteering fills community service requirements for schools and scouting organizations, Gantz says. For kids interested in science, participating gives the child or teen one-on-one time with a scientist or clinician, providing exposure to science they might not otherwise have.

“I really encourage other parents to find out what is happening in research at CHOP to see if their children qualify for a study,” Nolte says. “It can be very affirming and exciting to be part of research.”

New building has dedicated family research center

Children’s Hospital’s newest building, the Roberts Center for Pediatric Research, is now the site for research studies that don’t require a medical facility. If your family participated in research studies at 3535 Market St. in University City in the past, for the Center for Injury Research and Prevention (CIRP) or the Center for Autism Research (CAR), for example, those types of studies will now be conducted in a special area in the Roberts Center.

Families will be ushered to the Family Research Center on the fourth floor of the 21-story building. There, a concierge will greet you. There are waiting rooms, a lactation room, a family restroom and a pantry for families to use. Multiple evaluation rooms mean there is plenty of space for participants in CIRP, CAR or other groups’ studies.